RESUMEN
We analyzed the effect of hydroxyurea on the JAK2V617F allelic ratio (%JAK2V617F), measured in purified blood granulocytes, of patients with polycythemia vera and essential thrombocythemia. Thirty-six patients were examined sequentially prior to and after start of hydroxy-urea therapy (8 polycythemia vera, 17 essential thrombocythemia), or while remaining untreated (2 polycythemia vera, 9 essential thrombocythemia). Hydroxyurea therapy (median duration: 15 months) reduced the %JAK2V617F by >30% in 13/25 patients (4 polycythemia vera, 9 essential thrombocythemia). For 3 patients, JAK2V617F remained undetectable for 3-27 months. In addition, a single time point study of two large cohorts of patients, examined either at the time of diagnosis (99 polycythemia vera, 178 essential thrombocythemia) or while receiving hydroxyurea (36 polycythemia vera, 98 essential thrombocythemia; median length of therapy: 32 months), confirmed reduction of %JAK2V617F in the hydroxyurea-treated group (24% vs. 33% JAK2V617F at diagnosis, p<0.01). Prospective studies are needed to determine the prognostic value of reduced JAK2V617F allele burden under cytoreductive therapy.
Asunto(s)
Hidroxiurea/uso terapéutico , Janus Quinasa 2/genética , Policitemia Vera/tratamiento farmacológico , Trombocitemia Esencial/tratamiento farmacológico , Anciano , Anciano de 80 o más Años , Sustitución de Aminoácidos , Antidrepanocíticos/uso terapéutico , Estudios de Cohortes , Cartilla de ADN , Femenino , Granulocitos/fisiología , Humanos , Janus Quinasa 2/sangre , Janus Quinasa 2/deficiencia , Masculino , Persona de Mediana Edad , Mutación , Policitemia Vera/genética , Estudios Retrospectivos , Trombocitemia Esencial/genéticaRESUMEN
BACKGROUND: Few data are available on the efficacy of and tolerance to palifermin in the preventive treatment of oral mucositis in autograft patients treated with conditioning protocols usually used in France. METHODS: Our retrospective study bears on the first five candidates for autograft who benefited from prophylactic treatment with palifermin (60 microg/kg/day) between December 2005 and March 2006. RESULTS: Despite the prophylactic treatment, 3 patients developed severe oral mucositis. Moreover, in 3 patients, palifermin was found to be responsible for the onset of severe toxidermia, which required, in 2 cases, interruption of the treatment. CONCLUSION: These results need to be confirmed by a larger study that would also make it possible to evaluate the impact of palifermin on morbi-mortality in autograft patients in France, and to determine the safety profile of this drug.